{ "api_version": "1", "request": { "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sphingolipidoses&page=2", "query": { "condition": "Sphingolipidoses", "page": 2 }, "page_size": 10 }, "pagination": { "page": 2, "page_size": 10, "total_count": 10, "total_pages": 1, "next_page_url": null, "previous_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Sphingolipidoses&page=1&page_size=10" }, "source": "remote", "last_synced_at": "2026-05-22T08:45:29.010Z", "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.", "notes": [ "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.", "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.", "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees." ], "trials": [ { "nct_id": "NCT01839526", "title": "A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease", "overall_status": "TERMINATED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Fabry Disease" ], "interventions": [ { "name": "Iohexol", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Genzyme, a Sanofi Company", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "5 Years", "maximum_age": "25 Years", "sex": "MALE", "summary": "5 Years to 25 Years · Male only" }, "enrollment_count": 39, "start_date": "2013-05", "completion_date": "2016-09", "has_results": false, "last_update_posted_date": "2020-12-16", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 5, "location_summary": "La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more", "locations": [ { "city": "La Jolla", "state": "California" }, { "city": "Decatur", "state": "Georgia" }, { "city": "Cincinnati", "state": "Ohio" }, { "city": "Salt Lake City", "state": "Utah" }, { "city": "Fairfax", "state": "Virginia" } ], "official_url": "https://clinicaltrials.gov/study/NCT01839526" }, { "nct_id": "NCT02536911", "title": "A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1" ], "conditions": [ "Gaucher Disease" ], "interventions": [ { "name": "eliglustat", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Genzyme, a Sanofi Company", "sponsor_class": "INDUSTRY", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": "79 Years", "sex": "ALL", "summary": "18 Years to 79 Years" }, "enrollment_count": 24, "start_date": "2015-09", "completion_date": "2016-12", "has_results": false, "last_update_posted_date": "2017-02-10", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 2, "location_summary": "Miami, Florida • Knoxville, Tennessee", "locations": [ { "city": "Miami", "state": "Florida" }, { "city": "Knoxville", "state": "Tennessee" } ], "official_url": "https://clinicaltrials.gov/study/NCT02536911" }, { "nct_id": "NCT04252066", "title": "A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Fabry Disease" ], "interventions": [ { "name": "migalastat", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Amicus Therapeutics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": null, "sex": "FEMALE", "summary": "Female only" }, "enrollment_count": 20, "start_date": "2025-03-18", "completion_date": "2030-02", "has_results": false, "last_update_posted_date": "2025-07-08", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 1, "location_summary": "Philadelphia, Pennsylvania", "locations": [ { "city": "Philadelphia", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT04252066" }, { "nct_id": "NCT02785744", "title": "Genzyme Osteopenia/Osteoporosis Study", "overall_status": "COMPLETED", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Gaucher Disease" ], "interventions": [ { "name": "Gaucher disease DNA mutation analysis", "type": "OTHER" } ], "intervention_types": [ "OTHER" ], "sponsor": "NYU Langone Health", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 76, "start_date": "2016-04-01", "completion_date": "2019-09-01", "has_results": false, "last_update_posted_date": "2021-03-23", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 1, "location_summary": "New York, New York", "locations": [ { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT02785744" }, { "nct_id": "NCT04528355", "title": "Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC", "overall_status": "RECRUITING", "study_type": "OBSERVATIONAL", "phases": [], "conditions": [ "Primary Immunodeficiency (PID)", "Congenital Bone Marrow Failure Syndromes", "Inherited Metabolic Disorders (IMD)", "Hereditary Anemias", "Inflammatory Conditions" ], "interventions": [ { "name": "data collection", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Paul Szabolcs", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": "2 Months", "maximum_age": "60 Years", "sex": "ALL", "summary": "2 Months to 60 Years" }, "enrollment_count": 50, "start_date": "2020-08-20", "completion_date": "2028-06-30", "has_results": false, "last_update_posted_date": "2026-01-13", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 1, "location_summary": "Pittsburgh, Pennsylvania", "locations": [ { "city": "Pittsburgh", "state": "Pennsylvania" } ], "official_url": "https://clinicaltrials.gov/study/NCT04528355" }, { "nct_id": "NCT02583672", "title": "Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Gaucher Disease Type 1" ], "interventions": [ { "name": "N-acetylcysteine", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": true, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 33, "start_date": "2015-09", "completion_date": "2025-12-31", "has_results": false, "last_update_posted_date": "2026-02-18", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 2, "location_summary": "Minneapolis, Minnesota • New York, New York", "locations": [ { "city": "Minneapolis", "state": "Minnesota" }, { "city": "New York", "state": "New York" } ], "official_url": "https://clinicaltrials.gov/study/NCT02583672" }, { "nct_id": "NCT04693598", "title": "Gene Transfer Clinical Trial for Krabbe Disease", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Krabbe Disease" ], "interventions": [ { "name": "FBX-101", "type": "BIOLOGICAL" } ], "intervention_types": [ "BIOLOGICAL" ], "sponsor": "Forge Biologics, Inc", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "1 Day", "maximum_age": "12 Months", "sex": "ALL", "summary": "1 Day to 12 Months" }, "enrollment_count": 6, "start_date": "2021-11-05", "completion_date": "2026-11", "has_results": false, "last_update_posted_date": "2025-01-30", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 1, "location_summary": "Ann Arbor, Michigan", "locations": [ { "city": "Ann Arbor", "state": "Michigan" } ], "official_url": "https://clinicaltrials.gov/study/NCT04693598" }, { "nct_id": "NCT00813865", "title": "A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients", "overall_status": "COMPLETED", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Gaucher Disease", "Type 1 Gaucher Disease", "Gaucher Disease, Type 1" ], "interventions": [ { "name": "afegostat tartrate", "type": "DRUG" } ], "intervention_types": [ "DRUG" ], "sponsor": "Amicus Therapeutics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "18 Years", "maximum_age": null, "sex": "ALL", "summary": "18 Years and older" }, "enrollment_count": 8, "start_date": "2009-05-11", "completion_date": "2012-05-01", "has_results": true, "last_update_posted_date": "2018-08-15", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 3, "location_summary": "Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts", "locations": [ { "city": "Coral Springs", "state": "Florida" }, { "city": "Decatur", "state": "Georgia" }, { "city": "Boston", "state": "Massachusetts" } ], "official_url": "https://clinicaltrials.gov/study/NCT00813865" }, { "nct_id": "NCT02171104", "title": "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE2" ], "conditions": [ "Mucopolysaccharidosis Disorders", "Hurler Syndrome", "Hunter Syndrome", "Maroteaux Lamy Syndrome", "Sly Syndrome", "Alpha-Mannosidosis", "Fucosidosis", "Aspartylglucosaminuria", "Glycoprotein Metabolic Disorders", "Sphingolipidoses", "Recessive Leukodystrophies", "Globoid Cell Leukodystrophy", "Metachromatic Leukodystrophy", "Niemann-Pick B", "Niemann-Pick C Subtype 2", "Sphingomyelin Deficiency", "Peroxisomal Disorders", "Adrenoleukodystrophy With Cerebral Involvement", "Zellweger Syndrome", "Neonatal Adrenoleukodystrophy", "Infantile Refsum Disease", "Acyl-CoA Oxidase Deficiency", "D-Bifunctional Enzyme Deficiency", "Multifunctional Enzyme Deficiency", "Alpha-methylacyl-CoA Racmase Deficiency", "Mitochondrial Neurogastrointestingal Encephalopathy", "Severe Osteopetrosis", "Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)", "Inherited Metabolic Disorders" ], "interventions": [ { "name": "Stem Cell Transplantation", "type": "BIOLOGICAL" }, { "name": "IMD Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Only Preparative Regimen", "type": "DRUG" }, { "name": "Osteopetrosis Haploidentical Only Preparative Regimen", "type": "DRUG" }, { "name": "cALD SR-A (Standard-Risk, Regimen A)", "type": "DRUG" }, { "name": "cALD SR-B (Standard-Risk, Regimen B)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen C)", "type": "DRUG" }, { "name": "cALD HR-D (High-Risk, Regimen D)", "type": "DRUG" } ], "intervention_types": [ "BIOLOGICAL", "DRUG" ], "sponsor": "Masonic Cancer Center, University of Minnesota", "sponsor_class": "OTHER", "healthy_volunteers": false, "eligibility": { "minimum_age": null, "maximum_age": "55 Years", "sex": "ALL", "summary": "Up to 55 Years" }, "enrollment_count": 149, "start_date": "2014-07-10", "completion_date": "2029-07-14", "has_results": false, "last_update_posted_date": "2026-01-07", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 1, "location_summary": "Minneapolis, Minnesota", "locations": [ { "city": "Minneapolis", "state": "Minnesota" } ], "official_url": "https://clinicaltrials.gov/study/NCT02171104" }, { "nct_id": "NCT04127578", "title": "Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)", "overall_status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "phases": [ "PHASE1", "PHASE2" ], "conditions": [ "Parkinson Disease" ], "interventions": [ { "name": "LY3884961", "type": "BIOLOGICAL" }, { "name": "Methylprednisolone", "type": "DRUG" }, { "name": "Sirolimus", "type": "DRUG" } ], "intervention_types": [ "BIOLOGICAL", "DRUG" ], "sponsor": "Prevail Therapeutics", "sponsor_class": "INDUSTRY", "healthy_volunteers": false, "eligibility": { "minimum_age": "35 Years", "maximum_age": "80 Years", "sex": "ALL", "summary": "35 Years to 80 Years" }, "enrollment_count": 32, "start_date": "2020-01-03", "completion_date": "2030-12-31", "has_results": false, "last_update_posted_date": "2026-03-13", "last_synced_at": "2026-05-22T08:45:29.010Z", "location_count": 11, "location_summary": "Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more", "locations": [ { "city": "Scottsdale", "state": "Arizona" }, { "city": "Murrieta", "state": "California" }, { "city": "Englewood", "state": "Colorado" }, { "city": "Maitland", "state": "Florida" }, { "city": "Orlando", "state": "Florida" } ], "official_url": "https://clinicaltrials.gov/study/NCT04127578" } ] }